USA Patient Network Responds to Right To Try Legislation

Dear Members of the House Energy and Commerce Committee,

The USA Patient Network consists of patients, caregivers, and their friends and family members that are united by a common goal: to make sure that medical treatments are as safe, effective, and as affordable as possible. Unlike most patient groups, the USA Patient Network does not accept funding from pharmaceutical or medical device companies, or insurance companies. Representing individuals from across the United States, we are educating ourselves, and others, about the treatment standards that are most important to us, including improving quality of life, and the symptoms and health outcomes that matter most to patients. A major aspect of our work is to provide patients’ perspectives to federal agencies and medical and public health researchers.

We believe that there is no need for the proposed Right To Try Legislation (RTT). A pathway to such access already exists through the FDA’s Expanded Access program. Eligibility for patient participation falls into two groups defined by the FDA as 1) those with life-threatening diseases/conditions for whom “there is a reasonable likelihood that death will occur within a matter of months or in which premature death is likely without treatment,” and 2) those with serious diseases/conditions associated with morbidity that has a substantial impact on day to day functioning.” Of the over 1,000 expanded access requests received every year, the FDA approves 99.7%*, all while providing a level of protection for the patients that RTT does not. Those protections provide caps on costs and a reasonable assurance of benefit for the patient through prior Phase I and Phase II trials. The monitoring set in place under the current Expanded Access pathway will prevent untold harm by tracking serious and unexpected adverse events. Likewise, benefits are identified; guiding future advances in medical treatments.

Under the RTT legislation as currently proposed:

1. We cannot assume that untested drugs or devices will have any efficacy, or at worst won’t cause harm. We already know that there is an abundance of evidence that some FDA approved drugs have caused serious unforeseen harm. We can reasonably expect that the frequency and magnitude of harm from experimental drugs and devices will be unexpectedly and significantly greater.

2. It is understandable that a patient/family would be willing to try anything in an effort to find a solution for a terminal illness. However, does a terminal diagnosis mean that a patient loses all the ethical considerations and protections that we expect to be afforded to a patient with a non-terminal diagnosis?

We agree that patients who are terminally ill should have access to treatments, which could potentially be life-saving. While we recognize that desperate patients may be willing to take large risks to have such a chance, we also believe that those choices should be well informed of the risks and have the opportunity to participate in experimental treatments that are undergoing clinical trials. Giving patients hope without protections of clinical trials and reasonable assurances of safety as already demonstrated through Phase I and Phase II Clinical trials opens the door for unethical use of treatments. The RTT legislation does nothing to assure that desperate patients won’t be exploited or suffer even more painful deaths.

3. Unmonitored use of untested treatments may open the door to uses that otherwise might be illegal. Since there is no accountability or reporting required, there would be no way to track experimental use of treatments thus lacking any basis for safety. Without the oversight of the FDA and required reporting, protections from abusive use are absent. If the propelling reason behind RTT is to bring compassionate medical treatments to those who otherwise have no other option, then protections must be in place that will safeguard against misuse.

The system we currently have works, but if Congress is determined to pass legislation anyway, we strongly urge you to make the following amendments:

1. Add language to the legislation that mandates that companies or medical professionals cannot sell experimental treatments at a profit.

2. Require that all experimental treatments included in the legislation must be proven safe in completed Phase I or Phase II clinical trials conducted on at least 100 patients (not healthy volunteers).

3. Specify that all experimental drugs and devices available through RTT legislation should be in clinical trials regulated by the FDA.

4. Add language to mandate the reporting of harmful side effects and adverse events to the FDA by the physicians as well as drug or device companies.